Biogen Discontinues AAV Gene Therapy Work, Restructures Staff Amid Cost-Cutting Efforts

Biogen, a leading pharmaceutical company, has announced the complete discontinuation of its adeno-associated virus (AAV) based gene therapy programs, resulting in a significant staff reshuffle within the affected unit. This move comes as part of the company's broader cost-cutting initiative aimed at generating $1 billion in savings by 2025.

Staff Restructuring and Cost-Cutting Measures

Approximately 20 employees were impacted by the restructuring effort, with a majority being reassigned to other roles within Biogen. The company, which had over 7,600 employees worldwide at the end of 2024, has been implementing aggressive cost-cutting measures since July 2023. These measures include putting around 1,000 jobs on the chopping block to achieve the targeted savings.

Jane Grogan, Biogen's head of research, emphasized the company's disciplined approach to capital allocation. "We are taking a disciplined approach to capital allocation," Grogan stated, adding that Biogen will now focus its resources on "pioneer modalities" and therapies most likely to deliver "better outcomes for patients."

Industry-Wide Shift in AAV Gene Therapy

Biogen's decision to pull back from AAV-based gene therapies follows a similar move by Vertex Pharmaceuticals in May. Vertex had announced the end of its work on AAV capsids and terminated a gene editing agreement with Verve Therapeutics earlier this year.

The AAV platform has faced recent challenges, notably the controversy surrounding Sarepta Therapeutics' gene therapies. Sarepta reported three deaths this year: two associated with its Duchenne muscular dystrophy therapy Elevidys, and one linked to an investigational gene therapy for limb-girdle muscular dystrophy. All three deaths were attributed to liver failure, raising concerns about the safety of AAV-based gene therapies.

Diverging Views on AAV Technology

Despite the setbacks, some industry leaders remain optimistic about the future of AAV technology. Emil Kakkis, CEO of Ultragenyx, expressed confidence in AAV gene therapies during a recent interview. "I think AAVs are a very safe way to deliver DNA to the liver," Kakkis stated. "With continued improvement in AAV technology, it'll be around for a very long time."

Ultragenyx has reported success in testing AAV gene therapies for conditions such as Wilson disease and Sanfilippo syndrome, with "no safety issues" observed.

As the pharmaceutical industry continues to evolve, the future of AAV-based gene therapies remains uncertain. While some companies are stepping back from this technology, others see potential for improvement and long-term viability in treating various genetic disorders.